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Propylthiouracil

Propylthiouracil (PTU), also known as 6-propyl-2-thiouracil, is a thioamide-class antithyroid medication primarily used to treat hyperthyroidism by inhibiting the production of thyroid hormones in the thyroid gland. It is particularly indicated for managing Graves' disease, the most common cause of hyperthyroidism, and is preferred over alternatives like methimazole during the first trimester of pregnancy due to lower teratogenic risks. PTU is available only by prescription in oral tablet form (typically 50 mg) and requires regular monitoring of thyroid function to adjust dosing and prevent complications. PTU exerts its therapeutic effects through multiple mechanisms, including the inhibition of , an enzyme essential for incorporating iodide into residues within to form ( [T4] and [T3]). Additionally, it blocks the peripheral conversion of T4 to the more active T3, providing a rapid reduction in circulating thyroid hormone levels, which is crucial in acute settings like . This dual action distinguishes PTU from other antithyroid drugs and makes it a key option when immediate control of is needed, such as prior to or radioactive iodine ablation. For administration, adults typically receive an initial dose of 300 mg daily, divided into three doses every 8 hours, with maintenance therapy reduced to 100–150 mg daily once euthyroidism is achieved; children over 6 years may start at 5–7 mg/kg/day. In thyroid storm, a of 500–1,000 mg is often given, followed by 250 mg every 4 hours. Treatment duration varies but commonly lasts 12–18 months for , with beta-blockers sometimes co-administered to manage symptoms like . Despite its efficacy, PTU carries significant risks, including a warning for severe that can lead to , transplantation, or death, particularly in children, for whom it is not recommended except in rare cases. Other serious adverse effects include (0.2–0.5% incidence), necessitating patient education on symptoms like fever or , and antineutrophil cytoplasmic antibody (ANCA)-associated . Common side effects encompass , , and , while precautions include avoiding live , monitoring for bleeding due to potential , and using contraception during therapy due to potential fetal harm. Ongoing clinical surveillance, including complete blood counts and , is essential to mitigate these hazards.

Medical Uses

Indications

Propylthiouracil (PTU) is indicated for the management of in patients with or who are intolerant to methimazole and for whom or radioactive iodine is not an appropriate option, a condition characterized by excessive production of . In these scenarios, PTU inhibits thyroid hormone synthesis, helping to normalize thyroid function and alleviate symptoms such as , , and tremors. PTU is also utilized in the acute management of , a severe and potentially fatal complication of marked by multi-organ dysfunction, high fever, and cardiovascular instability. As a short-term intervention, it rapidly reduces circulating thyroid hormone levels, often in combination with supportive therapies, to stabilize patients during this endocrine emergency. It is indicated for pretreatment of patients prior to or radioactive iodine therapy in those intolerant to alternative agents, where PTU helps control symptoms and reduce risks. In comparison to methimazole, the other primary antithyroid drug, PTU demonstrates comparable efficacy in controlling but is preferentially selected in specific contexts, such as the first trimester of due to methimazole's higher association with congenital anomalies like aplasia cutis. Additionally, PTU is favored in because it not only blocks thyroidal hormone synthesis but also inhibits peripheral deiodination of thyroxine (T4) to the more potent (T3), providing a more comprehensive reduction in active hormone levels; however, a 2023 study found comparable outcomes with methimazole in critically ill patients, prompting calls for reevaluation of this preference.

Dosage and Administration

Propylthiouracil is available in oral tablet form, typically as 50 mg scored tablets. For adults with , the initial dose is usually 300 mg per day, administered orally in three divided doses at approximately 8-hour intervals; this may be increased to 400–600 mg daily for severe cases or large goiters, and occasionally up to 900 mg daily. Once euthyroid, the maintenance dose is typically reduced to 100–150 mg per day, divided into two or three doses, and adjusted based on clinical response. In pediatric patients, propylthiouracil is generally not recommended as first-line therapy due to the risk of severe , with methimazole preferred; it may be used in rare cases of methimazole intolerance when or radioactive iodine are not options. When indicated for children aged 6 years and older, a suggested initial dose is 50 mg daily, divided into three doses, with titration based on clinical response and . Therapy requires regular monitoring of thyroid function, including serum TSH and free T4 levels, every 4–6 weeks initially until stable, then every 2–3 months during maintenance to guide dose adjustments and ensure euthyroidism. The typical duration for inducing remission in hyperthyroidism is 12–18 months, after which the dose is tapered and discontinued if TSH and thyroid antibodies normalize; long-term low-dose therapy may be needed for persistent disease.

Pharmacology

Mechanism of Action

Propylthiouracil (PTU) primarily exerts its antithyroid effects by inhibiting the enzyme (TPO), a key component in the biosynthesis of . TPO catalyzes the oxidation of to iodine and facilitates the iodination of residues within , leading to the formation of monoiodotyrosine, diiodotyrosine, (T3), and thyroxine (T4). By binding to TPO, PTU blocks these processes, preventing organification and coupling reactions essential for thyroid hormone synthesis. The inhibition by PTU can occur through both reversible and irreversible mechanisms, depending on the iodide-to-drug ratio and reaction conditions. In scenarios with low concentrations relative to PTU, the drug leads to irreversible inactivation of TPO by forming reactive intermediates that covalently modify the , resulting in prolonged suppression of iodination. Additionally, PTU undergoes oxidation by TPO, producing metabolites such as and , which contribute to the overall inhibitory effect. This targeted action is enhanced by the accumulation of unmetabolized PTU in the thyroid gland following repeated administration, allowing for sustained local concentrations. Beyond its intrathyroidal effects, PTU uniquely inhibits the peripheral conversion of T4 to the more potent T3 by blocking the enzyme 5'-deiodinase in extrathyroidal tissues, such as the liver and . This dual mechanism—suppressing both synthesis and activation of —distinguishes PTU from methimazole, which primarily affects only thyroidal synthesis without significant peripheral deiodinase inhibition, making PTU particularly advantageous in acute conditions like where rapid reduction of circulating T3 is critical.

Pharmacokinetics

Propylthiouracil (PTU) is rapidly absorbed from the following , with estimated at 50-80% due to first-pass metabolism in the liver. Peak concentrations are typically achieved within 1 to 2 hours post-dose. The drug exhibits moderate , approximately 80%, primarily to and lipoproteins, with a of about 0.4 L/kg. PTU concentrates preferentially in the thyroid gland, reflecting its site of action, and it readily crosses the and enters . Metabolism occurs primarily in the liver through S-oxidation to form active intermediates, such as derivatives, followed by conjugation to glucuronides or inorganic sulfates. These hepatic processes contribute to the drug's short duration of action and its partial inhibition of peripheral thyroxine to conversion. Elimination is predominantly renal, with approximately 35% of the dose excreted in the within 24 hours as unchanged drug and conjugates; enterohepatic recirculation of metabolites prolongs its effective presence. The half-life is 1 to 2 hours and is not significantly altered by thyroid status, though it may be prolonged in hepatic or severe renal impairment. The relatively short half-life necessitates multiple daily dosing to maintain therapeutic levels, and dosage reductions may be warranted in hepatic dysfunction to avoid accumulation and toxicity; consult local guidelines for renal impairment.

Chemistry

Chemical Structure and Properties

Propylthiouracil, also known as 6-propyl-2-thiouracil, is an with the molecular formula C₇H₁₀N₂OS and a molecular weight of 170.23 g/mol. Its IUPAC name is 6-propyl-2-sulfanylidene-1H-pyrimidin-4-one. The molecule features a thiouracil core, consisting of a ring with a thioamide group at position 2 and a propyl attached at position 6, which contributes to its characteristic structure as a thioamide derivative. Physically, propylthiouracil appears as an odorless white crystalline powder. It has a of 219–221 °C. The compound is sparingly soluble in , with a solubility of approximately 1.2 mg/mL at 25 °C, but it dissolves more readily in alkaline solutions such as or hydroxides due to its weakly acidic nature. It is also soluble in and acetone but practically insoluble in nonpolar solvents like , , and . Propylthiouracil is sensitive to and oxidation, which can lead to degradation if not properly protected. It is incompatible with strong oxidizing agents and should be stored in tight, light-resistant containers at (preferably 15–30 °C) to maintain stability.

Synthesis

Propylthiouracil is synthesized through the of ethyl 3-oxohexanoate (also known as ethyl butyrylacetate) with under basic conditions. This method, a variant of the general for 6-substituted 2-thiouracils, was used in early preparations of the compound in the as part of efforts to develop more effective antithyroid agents beyond thiouracil. The reaction proceeds via and cyclization. First, the α-carbon of the β-ketoester is deprotonated by a base such as in to form an , which then attacks the electrophilic carbon of , yielding a thioamide intermediate. This intermediate undergoes intramolecular cyclization where the nitrogen attacks the ester carbonyl, followed by elimination of and tautomerization to the 2-thioxo-4-oxopyrimidine structure, incorporating the propyl chain at the 6-position. The mixture is then acidified to isolate the product. An alternative approach involves alkylation of 2-thiouracil with n-propyl iodide under basic conditions to introduce the side chain, though this typically targets N-alkylation rather than C-6 substitution and is less common for direct propylthiouracil production. A related route starts from ethyl acetoacetate and thiourea to form 6-methyl-2-thiouracil, followed by selective propylation, but this is not the primary method for the 6-n-propyl analog. In industrial production, an optimized one-pot process uses ethyl 3-oxohexanoate and with in at elevated temperatures (70–105°C), followed by acidification with HCl. Typical yields range from 70–80%, and the product is purified by and recrystallization from to achieve high purity.

Adverse Effects

Common Side Effects

Propylthiouracil commonly causes mild gastrointestinal side effects, including , , and epigastric distress or upset, which occur in up to 5% of patients at standard doses. These symptoms are typically self-limited and can be managed with dose adjustment or symptomatic therapies such as antacids. Dermatologic reactions are also frequent, encompassing skin rash, pruritus (itching), and urticaria, affecting approximately 4-5% of users. These cutaneous effects often appear early in treatment and usually resolve upon discontinuation or reduction of the drug. Mild hematologic changes, such as transient , may develop in less than 1% of patients and warrant routine monitoring of counts, particularly during the initial months of therapy. This is distinct from more severe and is often reversible with prompt intervention. Additional common adverse effects include (joint pain), , and temporary (alopecia), which are reported without precise incidence but are generally mild and resolve after treatment cessation. These effects tend to emerge within the first 3-6 months of propylthiouracil use and can often be addressed through dose reduction or supportive care.

Serious Adverse Effects

Propylthiouracil carries significant risks of severe , which can manifest as , , , or the need for transplantation, with fatalities reported in both adults and children. The U.S. (FDA) issued a black box warning in 2010 highlighting these risks, based on postmarketing reports of 13 cases of in adults (including five deaths) and additional pediatric cases, emphasizing that propylthiouracil should generally be reserved for patients intolerant to methimazole or when or radioactive iodine is not an option. Symptoms such as , anorexia, , , dark , or right upper quadrant pain necessitate immediate discontinuation and evaluation of , as the risk is highest within the first six months of therapy. The estimated incidence of severe hepatotoxicity is approximately 0.1-0.2%, though it may be higher in elderly patients or those on higher doses. Agranulocytosis represents another life-threatening adverse effect, characterized by severe ( <500/mm³), occurring in 0.2-0.5% of patients, most commonly within the first three months of treatment. Patients may present with fever, , , or signs of , requiring prompt drug discontinuation, white blood cell count monitoring, and supportive care to prevent or death. The risk appears dose-dependent and may be elevated in older adults or with higher initial doses. Propylthiouracil has also been associated with rare cases of , including (ANCA)-positive forms that can involve the kidneys (e.g., ), lungs (e.g., alveolar hemorrhage), or , with an incidence estimated at less than 0.1% (approximately 0.005-0.008% based on studies). These events, often occurring after months to years of , can be severe or and require immediate cessation of the drug upon suspicion, confirmed by ANCA testing and clinical evaluation. Postmarketing surveillance has further identified rare reports of (pancytopenia) and , which may overlap with and similarly demand assessment and drug withdrawal. Incidence rates for these hematologic effects are higher in vulnerable populations such as the elderly or those receiving high doses, underscoring the need for vigilant monitoring of complete blood counts.

Use in Pregnancy and Lactation

Propylthiouracil (PTU) is classified as FDA D, indicating positive evidence of human fetal risk based on adverse reaction data, but potential benefits may warrant use in certain cases of maternal . Although PTU carries a risk of congenital anomalies, including rare cases of aplasia cutis and other malformations such as urinary tract defects, this risk is lower compared to methimazole, making PTU the preferred antithyroid drug during the first trimester. Rare reports include two cases of exposure leading to severe and death in newborns. The American College of Obstetricians and Gynecologists (ACOG) and the American Thyroid Association (ATA) guidelines recommend PTU for treating in , with an initial dose of 300–600 mg/day divided into three doses, adjusted to the lowest effective level to maintain maternal euthyroidism while minimizing fetal exposure. To reduce the risk of fetal associated with PTU, guidelines advise switching to methimazole after the first (around 16 weeks ), provided maternal liver function is stable. Fetal monitoring is essential and includes serial ultrasounds to assess for goiter or growth restriction, particularly in the second and third , along with maternal every 4–6 weeks. Neonatal function should be evaluated shortly after birth to detect any transient or goiter resulting from transplacental drug transfer. During , PTU transfers into in low amounts, approximately 0.025% of the maternal dose (range 0.07-0.077%), and is considered compatible with when maternal doses are limited to 450 mg/day or less. The recommends monitoring the 's growth and development, with if clinical signs of appear, though routine testing is not required at low doses due to minimal impact on . No cases of significant adverse effects, such as liver damage, have been reported in breastfed infants exposed to PTU.

Special Considerations

Drug Interactions

Propylthiouracil (PTU) can potentiate the effects of oral anticoagulants such as by inhibiting K-dependent clotting factor synthesis, thereby increasing the risk of bleeding and requiring close monitoring of international normalized ratio (INR) levels. In patients with , PTU treatment leads to additive effects with beta-blockers and for symptom control, as normalization of function reduces the enhanced clearance of these drugs, potentially necessitating dosage adjustments to avoid or toxicity. PTU decreases clearance as resolves, elevating serum theophylline levels and increasing the risk of ; dose reduction of theophylline may be required with therapeutic monitoring. Concurrent administration of PTU with other antithyroid drugs, such as methimazole, should be avoided due to the risk of excessive hormone suppression leading to . High-iodine foods or supplements may partially counteract PTU's antithyroid effects by providing excess substrate for hormone synthesis despite PTU's inhibition of iodine organification, warranting dietary in iodine-replete patients. , due to its high iodine content, can reduce PTU's therapeutic efficacy and complicate management, necessitating careful of function during co-administration.

Contraindications

Propylthiouracil is contraindicated in patients with known to the drug or its components. It should be avoided in those with a history of serious adverse reactions, such as , , or like Stevens-Johnson syndrome, to PTU or other thionamide antithyroid drugs due to the potential for . PTU should generally be avoided in patients with active , including , or a history of PTU-induced due to the high risk of severe , which can progress to , transplantation, or death; alternative therapies like methimazole should be considered when clinically appropriate. During pregnancy, propylthiouracil is generally avoided in the second and third trimesters if methimazole is a viable alternative, as the latter carries a lower risk of hepatotoxicity while maintaining efficacy for hyperthyroidism management; however, propylthiouracil remains the preferred agent in the first trimester to minimize teratogenic risks associated with methimazole. Caution is advised with concurrent use of other agents known to cause bone marrow suppression, as this may increase the risk of severe hematologic toxicities like agranulocytosis; frequent monitoring of complete blood counts is recommended. In elderly patients, dose selection should be cautious due to potential decreased organ function and higher susceptibility to adverse effects, though no specific dose adjustment for renal impairment is required as PTU clearance is primarily non-renal.

Other Biological Effects

Role in Taste Perception

Propylthiouracil (PTU), chemically known as 6-n-propylthiouracil or PROP in taste research contexts, plays a significant role in elucidating human bitter taste perception through its interaction with genetic variants of the TAS2R38 bitter taste receptor gene. The TAS2R38 gene encodes a G-protein-coupled receptor that detects thiourea compounds like PTU, with common polymorphisms (such as the proline-alanine-valine or PAV haplotype for tasters and alanine-valine-isoleucine or AVI for non-tasters) determining an individual's sensitivity to PTU's bitterness. Individuals homozygous for the PAV variant (supertasters or medium tasters) perceive PTU as intensely bitter at low concentrations, while AVI homozygotes (non-tasters, approximately 25% of populations) detect little to no bitterness, and heterozygotes show intermediate sensitivity. This genetic basis positions PTU as a reliable phenotypic marker for classifying taste phenotypes in research, independent of its antithyroid properties. Studies utilize PTU-impregnated strips or solutions to categorize participants, revealing that exhibit heightened overall oral sensitivity, including to fats and other bitter compounds, which correlates with dietary preferences and behaviors. For instance, often report lower acceptance of and higher sensitivity to oral irritants. Population studies indicate variability in taster status ranging from 25% to 50% non-tasters across ethnic groups, highlighting PTU's utility in nutritional and food preference investigations. The mechanism underlying PTU's taste effects involves direct activation of and related type 2 taste receptors (T2Rs) expressed on fungiform and circumvallate papillae in the . Upon , PTU triggers a signaling cascade via gustducin, leading to increased intracellular calcium and of taste cells, which signals bitterness to the brain without involving thyroid hormone pathways. This peripheral receptor interaction explains why taste responses to PTU occur rapidly and are consistent across non-therapeutic dosing in sensory tests. In , PTU serves as a standard tool for screening preferences and sensitivities in product development and consumer studies, aiding in tailoring flavors to taster subgroups. Beyond research, clinical observations in patients receiving PTU therapy occasionally include reports of altered , such as loss of , unpleasant or metallic sensations, though these are infrequent and typically resolve upon discontinuation.

History

Development

Propylthiouracil (PTU) was developed as part of early research into antithyroid compounds, inspired by observations of goitrogenic effects in rats fed diets containing seeds, such as those from and , reported by Kennedy and Purves in 1941. These findings highlighted natural substances capable of inducing enlargement by interfering with hormone synthesis, prompting further investigation into synthetic analogs. Edwin B. Astwood, working at , initiated screening of derivatives for similar goitrogenic activity in rats starting in 1941, building on concurrent reports by C. G. and J. B. MacKenzie that sulfaguanidine and caused hyperplasia in rodents. This systematic approach aimed to identify compounds that could safely inhibit function, offering potential therapeutic alternatives to surgical interventions for . Preclinical testing confirmed PTU's potency as a through Astwood's publication in the Journal of Pharmacology and Experimental Therapeutics, where he detailed the chemical structures of effective -based compounds, including early thiouracil variants, and their ability to block iodine incorporation into in rat thyroids. These studies demonstrated that PTU and related derivatives were far more active than parent , with PTU specifically showing strong goitrogenic effects at low doses without excessive toxicity in animal models. The rationale emphasized developing safer agents than initial thiouracil, which exhibited concerning side effects like granulocytopenia in preliminary trials. By , Astwood and W. P. VanderLaan reported in the Journal of Clinical Endocrinology and Metabolism on optimized thiouracil derivatives, identifying 6-n-propylthiouracil (PTU) as particularly effective due to its enhanced activity-toxicity ratio compared to thiouracil. PTU was named for its structural features: the n-propyl substituent at the 6-position of the thiouracil base, which improved its pharmacological profile. Early challenges included addressing the leukopenic risks observed with thiouracil, leading to PTU's refinement as a less toxic option through iterative synthesis and testing in collaboration with pharmaceutical firms like . These efforts culminated in PTU's recognition as a viable by the mid-1940s, though no individual was filed by Astwood, with production handled by industry partners.

Clinical Adoption and Guidelines

Propylthiouracil (PTU) received FDA approval in 1947 for the treatment of , with initial clinical application focused on managing . By the 1950s, PTU had achieved widespread adoption as a cornerstone antithyroid therapy, supplanting earlier surgical and radioactive iodine approaches in many settings due to its efficacy in inhibiting thyroid hormone synthesis. Key regulatory and guideline milestones shaped PTU's clinical trajectory. In 2010, the FDA issued a black box warning highlighting the risk of severe , including and death, based on post-marketing surveillance identifying 13 cases of PTU-induced in children under 18 between 2004 and 2009. The 2016 American Thyroid Association (ATA) guidelines recommended methimazole as the preferred first-line antithyroid drug for most adults with , reserving PTU primarily for the of or cases of methimazole intolerance due to PTU's superior safety profile in early gestation despite its hepatotoxicity risks. A 2017 survey of clinical endocrinologists found that 98% preferred methimazole over PTU (2%) as initial therapy for non-pregnant adults with . Globally, PTU has been included on the World Health Organization's Model List of since 1977, underscoring its role in resource-limited settings for thyrotoxicosis management. However, its usage has declined in favor of methimazole over the past two decades, driven by PTU's higher incidence of serious adverse effects. As of 2025, PTU's adoption reflects heightened caution amid reports of (ANCA)-associated , with multiple cases documented post-2020 linking long-term use to severe manifestations such as alveolar hemorrhage and renal involvement, including cases reported in 2025, further contributing to reduced prescribing. No major global guideline updates have altered core recommendations since the 2016 ATA guidelines, though regional updates such as the 2025 Korean Thyroid Association guidelines address specific management aspects like PTU discontinuation prior to scans. PTU remains societally impactful in emergency care, serving as a key agent for management due to its dual action in blocking hormone synthesis and peripheral T4-to-T3 conversion, with guidelines endorsing high-dose regimens (600–1,000 mg loading followed by 200–250 mg every 4–6 hours) in critically ill patients. As a generic available since the 1950s, PTU ensures broad accessibility, particularly in acute hyperthyroid crises where rapid intervention is essential.